Antisense therapy, a magic bullet for the treatment of various diseases: Present and future prospects

Abstract

Antisense oligonucleotides (ASO) are becoming more widely accepted as potential therapeutics for various diseases. Antisense therapy has emerged as an exciting and promising strategy for the treatment of various diseases. Antisense are different from conventional drugs that are designed to interact with protein molecule. Antisense drugs have more selective action and they have the potential to be more effective and less toxic than conventional drugs. Recent clinical trials confirm the ability of antisense to significantly suppress target-gene expression. Antisense oligonucleotides are usually highly selective and thus produce fewer adverse effects than conventional therapeutics. Antisense technology might be a gateway to the treatment of diseases by targeting the expression of genes rather than permanently altering them. Antisense oligonucleotides can alter target gene expression by binding to RNA. Once bound, the ASO either disables or induces the degradation of the target RNA. This technology may be used to treat various conditions including cancer, diabetes, and hypertension, as well as autoimmune and cardiovascular diseases. ASOs are potentially potent, selective and well-tolerated drugs. The current status and future direction of several antisense drugs that have potential clinical use in treatment of various diseases are reviewed here. Key words: Antisense, oligonucleotide, gene, autoimmune disease, degradation.